141 completed, 40 terminated, 36 active not recruiting, 24 recruiting. 281 Phase 3, 74 Phase 2.
Follow Heredodegenerative Disorders, Nervous System: new trials by email Literature Review of Heredodegenerative Disorders, Nervous System trials Research Report on Heredodegenerative Disorders, Nervous System
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome NTI164, Placebo - RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome | Phase 2 / Phase 3 | Not yet recruiting | Fenix Innovation Group | 40 | 2027-07-01 | 2026-09-09 |
| A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS Codabakalner, Placebo - Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD) | Phase 2 / Phase 3 | Not yet recruiting | Spinogenix | 120 | 2026-12-01 | 2026-10-09 |
| Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3 PLX-200, Placebo - Juvenile Neuronal Ceroid Lipofuscinosis | Phase 3 | Withdrawn | Polaryx Therapeutics, Inc. | 2026-11-01 | 2026-08-19 | |
| To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) Acoramidis (AG10) - Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial | Phase 3 | Not yet recruiting | Eidos Therapeutics, a BridgeBio company | 250 | 2026-11-01 | 2026-08-11 |
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia Nomlabofusp, Placebo - Friedreich Ataxia, Friedreich's Ataxia | Phase 3 | Recruiting | Larimar Therapeutics, Inc. | 150 | 2026-10-26 | 2026-10-09 |
| To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome Bionetide, Placebo - Rett Syndrome | Phase 3 | Recruiting | Biomed Industries, Inc. | 210 | 2026-09-30 | 2026-03-27 |
| Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients. TETA 4HCl formulation, D-Penicillamine - Wilson's Disease | Phase 3 | Recruiting | Orphalan | 38 | 2026-09-03 | 2026-10-15 |
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 70 | 2026-09-01 | 2026-10-13 |
| Everolimus bAsed caLcineurin inhibiTor frEe immunosuppRession oNe Year AfTer lIver transplantatiON (ALTERNATION) Everolimus, Tacrolimus or Cyclosporine - Liver Allograft, Liver Diseases | Phase 3 | Recruiting | Hannover Medical School | 150 | 2026-08-19 | 2026-10-05 |
| Pivotal Study of N-acetyl-L-leucine for CACNA1A N-Acetyl-L-Leucine, Placebo - CACNA1A, Spinocerebellar Ataxia Type 6 | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2026-08-17 | 2026-11-03 |
| Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Vatiquinone - Friedreich's Ataxia | Phase 3 | Not yet recruiting | PTC Therapeutics | 120 | 2026-08-15 | 2026-08-14 |
| A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1) Rinzimetostat, Darolutamide, Enzalutamide - Metastatic Castration Resistant Prostate Cancer | Phase 3 | Recruiting | ORIC Pharmaceuticals | 600 | 2026-08-03 | 2026-11-03 |
| FOLFIRI Plus Bevacizuamb and QL1706 in Second-Line Treatment for mCRC FOLFIRI + bevacizumab + QL1706, FOLFIRI + bevacizumab - Colorectal Cancer Metastatic, Microsatellite Stable (MSS) Colorectal Cancer (CRC) | Phase 3 | Not yet recruiting | Sun Yat-sen University | 270 | 2026-07-30 | 2026-07-27 |
| A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome fenfluramine hydrochloride, Placebo - Rett Syndrome | Phase 3 | Recruiting | UCB BIOSCIENCES, Inc. | 200 | 2026-07-14 | 2026-11-02 |
| [64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy [64Cu]FBP8 PET/MR - Cardiac Amyloidosis | Phase 3 | Recruiting | Brigham and Women's Hospital | 20 | 2026-07-07 | 2026-07-21 |
| Pridopidine Phase 3 Study in Huntington's Disease Pridopidine, Placebo - Huntington Disease | Phase 3 | Recruiting | Prilenia | 400 | 2026-07-01 | 2026-11-04 |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) Zeleciment Rostudirsen (DYNE-251), Placebo - Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne | Phase 3 | Recruiting | Dyne Therapeutics | 90 | 2026-06-01 | 2026-05-27 |
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 zeleciment basivarsen (DYNE-101), Placebo - Myotonic Dystrophy Type 1 (DM1), DM1 | Phase 3 | Recruiting | Dyne Therapeutics | 150 | 2026-05-14 | 2026-11-06 |
| Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome TSHA-102 - Rett Syndrome | Phase 3 | Active not recruiting | Taysha Gene Therapies, Inc. | 4 | 2026-05-08 | 2026-08-19 |
| SCRT Based iTNT vs. LCRT Based TNT for MSS Locally Advanced Rectal Cancer short-course radiaotherapy, long-course chemoradiaothearpy, Capecitabine - Locally Advanced Rectal Cancer (LARC), MSS (Microsatellite Stable) | Phase 3 | Not yet recruiting | Fudan University | 612 | 2026-05-01 | 2026-04-24 |
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Huntington Disease (29) · Amyloid Neuropathies, Familial (22) · Tourette Syndrome (21) · Hereditary Sensory and Autonomic Neuropathies (20) · Mucopolysaccharidosis II (19) · Tuberous Sclerosis (19) · Amyloidosis (18) · Rett Syndrome (18) · Myotonic Dystrophy (17) · Friedreich Ataxia (16) · Fragile X Syndrome (13) · Sudden Infant Death (12) · Nervous System Diseases (11) · Optic Atrophy, Hereditary, Leber (11) · Spinocerebellar Ataxias (11)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (278) · Genetic Diseases, Inborn (272) · Nervous System Diseases (270) · Neurodegenerative Diseases (217) · Central Nervous System Diseases (136) · Brain Diseases (119) · Neurologic Manifestations (101) · Nutritional and Metabolic Diseases (95) · Metabolic Diseases (92) · Neuromuscular Diseases (85) · Mental Disorders (68) · Genetic Diseases, X-Linked (65) · Congenital Abnormalities (63) · Pathological Conditions, Signs and Symptoms (63)
Aripiprazole (7) · Everolimus (6) · Sirolimus (6) · deutetrabenazine (6) · idursulfase (6) · Cannabidiol (5) · Zolgensma (5) · attruby (5) · idebenone (5) · Mexiletine (4) · tafamidis (4) · tanezumab (4) · trofinetide (4) · BPN14770 (3) · Riluzole (3)
Novartis Pharmaceuticals (14) · Assistance Publique - Hôpitaux de Paris (8) · Pfizer (8) · Eidos Therapeutics, a BridgeBio company (6) · Kissei Pharmaceutical Co., Ltd. (6) · Jazz Pharmaceuticals (5) · Nobelpharma (5) · Otsuka Pharmaceutical Development & Commercialization, Inc. (5) · Santhera Pharmaceuticals (5) · Teva Branded Pharmaceutical Products R&D, Inc. (5) · ACADIA Pharmaceuticals Inc. (4) · GenSight Biologics (4) · JCR Pharmaceuticals Co., Ltd. (4) · Lupin Ltd. (4) · Shire (4)
United States (172) · United Kingdom (85) · France (83) · Spain (81) · Canada (78) · Germany (78) · Italy (77) · Australia (59) · Netherlands (44) · Belgium (39) · Brazil (36) · Japan (36) · Poland (30) · South Korea (26) · Denmark (23)
Eva Kohegyi, MD (5) · Paul Wang, M.D. (3) · Thomas Klopstock, MD (3) · David Lynch, MD (2) · David Nguyen, PhD (2) · Elizabeth Berry-Kravis, MD (2) · Elliott Vichinsky, MD (2)
PMID 30936738, PMID 36449262, PMID 37460385, PMID 33395098, PMID 34108929, PMID 37460782, PMID 21779958, PMID 26554876, PMID 26666500, PMID 27494299, PMID 28265459, PMID 29023494, PMID 30053159, PMID 30558645, PMID 31353964, PMID 31937586, PMID 32107748, PMID 32524297, PMID 33070419, PMID 33461041, PMID 34115419, PMID 34355354, PMID 34383289, PMID 34923848, PMID 34957550, PMID 35747889, PMID 35799473, PMID 35961250, PMID 36715498, PMID 37434378, PMID 37915038, PMID 37943223, PMID 38548524, PMID 38557959, PMID 38723617, PMID 38932583, PMID 39383458, PMID 39825184, PMID 40488446, PMID 41205147, PMID 42389794, PMID 42658466, PMID 1463007, PMID 15492353, PMID 15965209, PMID 1674640, PMID 18441370, PMID 18441371, PMID 18678823, PMID 18774912
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